3 results
Approved WMOCompleted
Primary:To assess the safety and tolerability of Firdapse after single and multiple doses Secondary:Part 1 only: To determine the dose-related PK profile of single, escalating doses of Firdapse;Part 2 only: To assess the steady-state plasma PK and…
Approved WMOCompleted
The overall objective of this study is to evaluate the efficacy and safety of neoGAA in treatment-naïve patients with LOPD as compared to alglucosidase alfa, when this is administered biweekly for a period of 49 weeks. Also, there is an open-label…
Approved WMOCompleted
Feasibility is assessed after every VR-EMDR session and after treatment completion. PTSD symptoms, quality of life and self-empowerment are assessed before and after treatment.